- A phase 2 clinical trial to test the experimental drug GM604 in people with ALS has opened at two trial sites in the United States.
- The study, which is expected to enroll 12 adults with ALS, is designed to test the safety and tolerability of GM604.
Update (May 12, 2014): Genervon announced that preliminary results of this phase 2a trial of GM604 show "significant promise for treating ALS." Full results are expected later in 2014. In an April 28, 2014, press release, the company said seven of the eight trial participants who received treatment with GM604 experienced slowing or stopping of functional deterioration 10 weeks after completion of two weeks of intravenous infusions of the drug (without further treatment). Genervon says it is planning the next trial for GM604 and intends to partner with one or more pharmaceutical companies on this.
Update (March 3, 2013): This study is no longer recruiting new participants.
Update (July 1, 2013): Genervon announced in a June 26, 2013, press release, that the U.S. Food and Drug Administration (FDA) has granted fast track designation to its experimental drug GM604. Fast track designation allows for faster FDA review of drugs that treat serious diseases and fill an unmet need.
A phase 2 clinical trial to test the experimental drug GM604 in people with amyotrophic lateral sclerosis (ALS) has opened at two trial sites in the United States. Enrollment is expected to begin soon.
GM604 technology, developed by Genervon Biopharmaceuticals in Pasadena, Calif., is based on "master regulators," which are molecules found in early-stage human development that control the development of the human nervous system.
According to Genervon, the GM604 molecule's structure is identical to one of nine active parts of a neurotrophin called motoneuronotorphic factor (MNTF). The company says it confers neuroprotection via control and regulation of numerous ALS-associated genes and pathways.
The study, which is expected to enroll 12 adults with ALS, is designed to test the safety and tolerability of GM604. Additional study objectives include determining preliminary effects of GM604 on ALS disease biomarkers and other measures of disease progression.
How to participate in the phase 2 trial
Prospective trial participants must be at least 18 years old and meet the El Escorial criteria for a definite diagnosis of ALS. In addition, they must:
- be able to comply with study procedures;
- be medically cleared to undergo lumbar (lower back) puncture for the collection of cerebrospinal fluid;
- not have a history of liver disease, severe renal (kidney) failure, diabetes, coronary heart disease or cancer; and
- meet additional criteria.
Study site locations are:
Massachusetts General Hospital (not yet recruiting)
Principal investigator: Nazem Atassi
Sub-investigator: Merit Cudkowicz
Contact: Michele Parkinson, 617-643-6249, firstname.lastname@example.org
Columbia Medical Center (not yet recruiting)
Principal investigator: Hiroshi Mitsumoto
Contact: Nicole Armstrong, 212-305-8148, email@example.com
To learn more, see GM604 Phase 2A Randomized Double-Blind Placebo-Controlled Pilot Trial in Amyotrophic Lateral Disease (ALS) (GALS), or search for trial ID NCT01854294 at ClinicalTrials.gov.
About Clinical Trials
A clinical trial is a test, in humans, of an experimental treatment. Although it's possible that benefit may be derived from participating in a clinical trial, it's also possible that no benefit, or even harm, may occur.
MDA has no ability to influence who is chosen to participate in a clinical trial.
To learn more, see Learn About Clinical Studies and Being a Co-Adventurer, which is about neuromuscular disease clinical trials. To see a continuously updated database of clinical trials, go to ClinicalTrials.gov.